Drugs / PBFT02
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04747431 | Sep 2021 → Aug 2028 expected | GRN-related frontotemporal lobar degeneration with Tdp43 inclusions, frontotemporal dementia and/or amyotrophic lateral sclerosis 1 | Passage Bio, Inc. | Active not recruiting | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | PBFT02 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04747431 ↗ |
| Action | Restore | “deliver a functional copy of the GRN gene to the brain” NCT04747431 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “adeno-associated viral vector serotype 1 carrying GRN” NCT04747431 ↗ |
| Route | Other | “via intra cisterna magna” NCT04747431 ↗ |
| Target | GRN | “deliver a functional copy of the GRN gene to the brain” NCT04747431 ↗ |